CER Daily Newsfeed

CMS Releases Final Guidance for 2028 Medicare Drug Price Negotiation Cycle

(10/6, Joanne Walker, The Evidence Base) reports “...The White House announcement highlighted three provisions. First, the Orphan Drug Exclusion has been broadened to cover products designated by the FDA for one or more rare diseases, provided all approved indications are for such conditions. This preserves incentives for rare disease research while maintaining negotiation eligibility. Recent analysis from the National Pharmaceutical Council suggests the IRA’s original exclusion may have discouraged development across multiple rare diseases, with second orphan designations falling by nearly 50% after the law’s passage. The broadened exclusion may help to ease these and other concerns.” Full

Real-World Findings on Incidence of Adverse Events Highlighted for JAK Inhibitors

(10/6, Tim Smith, HCPLive) reports “Real-world data suggest Janus kinase (JAK) inhibitors, especially upadacitinib, baricitinib, and abrocitinib, have a low incidence of serious adverse events in patients with atopic dermatitis and alopecia areata...‘Considering that several real-world studies focusing on oral JAK inhibitors for the treatment of inflammatory skin diseases have been published in recent years, this study aims to explore the safety of oral JAK inhibitors with inflammatory skin diseases in real-world patients by conducting a meta-analysis of real-world studies,’ Gao et al wrote.” Full

Outcomes Model Is Starting With Gene Therapy for Sickle Cell Disease

(10/3, Ryan Flinn, Managed Healthcare Executive) reports “...The Cell and Gene Therapy Access Model, launched by the CMS in July, allows CMS to negotiate outcomes-based contracts with drugmakers on behalf of states. So far, 33, along with Washington, D.C., and Puerto Rico, have joined the program. As part of the agreement, drugmakers will be required to provide rebates to states if the therapies fail to deliver their expected clinical benefits. The program is initially focusing on treatments for sickle cell disease...although CMS is exploring expanding the program into different conditions and therapies in the future.” Full

Another Drug Pricing Demo Surfaces After Trump-Pfizer Deal

(10/3, Maaisha Osman, Inside Health Policy) reports “...The Trump administration's Spring Unified Agenda had said two forthcoming CMS Innovation Center drug pricing model rules were in the works but provided no details, and McDermott analysts tell Inside Health Policy the GLOBE and GUARD models likely correspond to those entries.” Subscription Required

Real-World Evidence Shapes Therapy for Rare Lung Cancer

(10/5, European Medical Journal) reports “Immunotherapy may offer meaningful benefit for patients with combined small-cell lung cancer, a rare and aggressive tumor subtype that has historically lacked clear treatment strategies. A real-world analysis of 30 patients has provided new insights into clinicopathological features and outcomes, suggesting that immune checkpoint blockade could play a valuable role in disease control.” Full